Australian cancer researchers have successfully developed a next-generation gene-editing tool to enhance the study of such dangerous human diseases. With this tool, scientists hope to gain a deeper understanding of this deadly illness and develop effective treatment methods, as reported by Medical Xpress.
Researchers from the Olivia Newton-John Cancer Research Institute and their collaborators created a gene-editing model using an advanced version of the Cas12a enzyme. This breakthrough allows for more precise genetic studies and brings gene-editing methods closer to real-world applications, they stated.
Gene editing functions like molecular scissors, enabling scientists to cut and modify DNA—the genetic instructions that control cell behavior and are responsible for many diseases and other traits we exhibit.
CRISPR, widely used in medical research and gene editing, typically relies on an enzyme called Cas9. While Cas9 has been the standard for the past decade, Cas12a offers a key advantage: it can remove multiple genes simultaneously with greater precision. The study's author, Dr. Eddy La Marca, explained, "For the first time, Cas12a has been used in preclinical models, which could significantly expand our capabilities in genomic engineering."
After testing the new tool on lymphoma, a type of blood cancer, the team identified genes that caused the disease to progress more rapidly. They also combined Cas12a with other editing methods, allowing them to make more complex changes to DNA. Researchers Wei Jin and Yexuan Deng reported that they also crossed the Cas12a animal model with a model expressing a modified version of Cas9, enabling them to simultaneously delete and activate different genes. This will allow researchers to use this tool for modeling and studying complex genetic diseases, the authors believe.
Professor Marco Gerold, one of the leading researchers, believes this discovery will aid scientists worldwide. "We are confident that this work will encourage other research groups to use the Cas12a preclinical model, which, when combined with screening libraries, represents a new powerful toolkit for gene editing that can enhance our understanding of the mechanisms underlying many types of cancer," he stated.
The ability to edit multiple genes simultaneously could pave the way for new treatments for serious diseases. As gene editing becomes increasingly integrated into real medical procedures, researchers are also working on safe ways to introduce these tools into patients' bodies. Professor Gerold added, "This Cas12a preclinical model will also contribute to our understanding of how CRISPR tools can be more effectively translated into clinical practice." The authors aim to ensure the safety and efficacy of this technology for future treatment methods that utilize it.
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